Tuesday, April 14, 2009

Our Drew lost his battle with ALS on Tuesday April 7, 2009. His family would like to thank all of the people who made Drew's journey a little more bearable by following his blog, leaving notes of encouragement and comments. He will not be forgetten.

Wednesday, March 11, 2009

Some states push back against stem cell researchBy SHANNON McCAFFREY, Associated Press Writer Shannon Mccaffrey, Associated Press Writer 1 hr 31 mins agoATLANTA – A showdown is shaping up in some of the nation's most conservative states over embryonic stem cell research, as opponents draw language and tactics from the battle over abortion to counter President Barack Obama's plan to ease research restrictions.Legislation granting fertilized embryos "personhood" has gained momentum in at least three state legislatures. The strategy — which has been used to try to undermine the Roe v. Wade decision legalizing abortion — is now aimed at embryonic stem cell research. The scientific field uses stem cells from human embryos, which can develop into different kinds of adult cells, to seek answers about human health.Opposition to both abortion and stem cell research hinges on the same issue: When does life begin? As a result, embryonic stem cell research has become the latest front in the decades-old battle over abortion."If you are someone who believes that a single cell embryo is a person then you are looking for any opportunity you can to make that argument. But as a country, legally, we've never accepted that," said Michael Werner of the Coalition for the Advancement of Medical Research. "The legislative tactics are the same."Abortion opponents believe embryonic stem cell research is an assault on life in its earliest form. Fertilized embryos are destroyed when stem cells are extracted from them for research."No one's right for a cure supersedes someone else's right to life," said Dan Becker, president of Georgia Right to Life.The opponents expect to push for restrictions in conservative-leaning states. And they say states must take the lead in pushing the abortion and stem cell issues into the increasingly conservative federal courts.Legal experts said the state measures restricting stem cell research raise constitutional concerns in a largely untested area of law.Alta Charo, professor of law and bioethics at the University of Wisconsin, said a new line of legal thought holds that scientific inquiry should be protected by the First Amendment, "like a political or religious statement or activity."She said the measures restricting the use of fertilized embryos also raise questions about the right to procreation."The courts haven't settled this yet," Charo said.While Louisiana already bans the destruction of fertilized embryos, the courts have not yet weighed in, Charo said.In Georgia, a measure that would ban some forms of stem cell research on fertilized embryos is moving quickly through the state Senate. The bill would outlaw the destruction of fertilized embryos, which the legislation defines as a person. It is expected to face a vote in the full state Senate on Thursday.Similar "personhood" measures have cleared one chamber each in Montana and North Dakota.They come in the wake of a Colorado ballot initiative that said human life begins at conception. It failed to win voter approval last year.David Prentice, senior fellow for life sciences at the Washington, D.C.-based Family Research Council, said Obama's announcement Monday that he will free federal funds for embryonic stem cell research will rally conservatives."This is the beginning," Prentice said. "I think there will be more to come."In 2001, President George W. Bush limited federal funding for embryonic stem cell research to 21 stem cell lines already in existence. Because they were already being used for research, Bush allowed work on them to continue.Obama's new approach will enable federally funded researchers to use hundreds of new embryonic stem cell lines. Supporters believe the research could lead to treatments for major disorders, such as Parkinson's disease and spinal injuries.Eight states bucked the Bush administration limits and allowed state money to be spent on the research: California, Connecticut, Illinois, Iowa, Maryland, Massachusetts, New Jersey and New York. Some of them, struggling with gaping budget deficits, may rein in state funding for those research programs, now that federal dollars will again be flowing.Sean Tipton, director of public affairs at the American Society for Reproductive Medicine, said legislation that would affect stem cell research has been introduced in several states, including Alabama, Georgia, Maryland, Montana, North Dakota and South Carolina."It's clearly part of a national strategy and at some point it will probably succeed," Tipton said.Tipton said advocacy groups are targeting states where they have the best chance of success.One of those is Georgia, where Gov. Sonny Perdue has said he opposes embryonic stem cell research, even as he tries to lure biotech companies to state."I am absolutely opposed to creating embryos to cure a disease," Perdue told reporters this week.The Georgia bill cleared the Senate Health and Human Services Committee by a close 7-6. The religious conservatives pushing it are influential with Georgia's Republican-led Legislature.Opponents say the Senate bill would be a blow to the state's thriving research universities, as well as fertility clinics that perform thousands of in-vitro treatments every year."We have the president of the United States saying he is going to put science ahead of politics and unfortunately in Georgia we are moving in the opposite directions," said state Sen. David Adelman, a Democrat from Decatur.___National Conference of State Legislatures Genetic Technologies Project: http://www.ncsl.org/programs/health/genetics

European Commission Awards Orphan Designation to Knopp Neurosciences' KNS-760704 in ALSPITTSBURGH--(BUSINESS WIRE)--Knopp Neurosciences Inc. ("Knopp") today announced that the European Commission ("EC") has designated KNS-760704 as an orphan medicinal product for the treatment of Amyotrophic Lateral Sclerosis.The EC decision, based on a favorable opinion from the European Medicines Agency (EMEA), follows the designation of KNS-760704 as an orphan drug for the treatment of ALS by the U.S. Food and Drug Administration in 2007. KNS-760704 is currently in Phase 2 clinical trials for ALS, a universally fatal neurodegenerative disease with limited treatment options."We are very pleased with the European Commission's designation of KNS-760704 as an orphan medicinal product and the recognition of its potential to be of significant benefit for patients with ALS," said Michael Bozik, M.D., president and CEO of Knopp. "Orphan medicinal product designation will significantly facilitate our efforts to develop a safe and effective treatment for patients suffering from this relentless disease."To stimulate the research and development of orphan drugs, the European Union ("EU") has established a centralized procedure for the designation of orphan medicinal products and provides incentives for the development of medicinal products for rare disorders. Companies with an orphan designation for a medicinal product benefit from incentives that include fee reductions, a 10-year market exclusivity period following authorization for designated products, scientific advice to optimize development, and direct access to the EMEA centralized procedures for marketing authorization.Knopp plans to initiate Phase 3 development of KNS-760704 in ALS in late 2009.About KNS-760704KNS-760704 is a low molecular weight benzothiazole shown to improve mitochondrial function and confer significant cellular protection in neurons under stress. The chirally pure form of the synthetic benzothiazole (6R)-2-amino-4,5,6,7-tetrahydro-6-(propylamino)benzothiazole, KNS-760704 is highly orally bioavailable, water soluble, renally excreted, and only moderately protein bound. In Phase 1 studies, the compound was shown to be safe and well tolerated in healthy human subjects. Phase 2 studies of KNS-760704 in ALS are ongoing. The compound has received orphan drug designation from the U.S. Food and Drug Administration and the European Commission for the treatment of patients with ALS.About ALSAmyotrophic lateral sclerosis, also known as Lou Gehrig's disease and Charcot's sclerosis, is a rapid, universally fatal neurodegenerative disorder characterized by progressive muscle weakness and wasting. ALS affects adults in the prime of life and creates a substantial burden for caregivers. U.S. prevalence is approximately 20,000 and the global incidence is approximately two per 100,000. Only one drug has been approved for the treatment of ALS. Life expectancy after symptom onset is usually three to five years.About Knopp Neurosciences Inc.Knopp Neurosciences is a drug discovery and development company focused on delivering breakthrough treatments for neurological disorders through innovation, experience, and partnership. The company's lead product candidate is KNS-760704, an orally bioavailable small molecule in development for the treatment of ALS. Knopp's leadership includes experienced neuroscience drug development and discovery executives formerly associated with major pharmaceutical companies. Knopp's financing has been led by Saturn Capital Inc. of Boston as placement agent and Saturn Partners II as lead funder.This press release contains "forward-looking statements," including statements relating to Knopp's planned regulatory filings and clinical development programs for KNS-760704. All forward-looking statements are based on management's current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including the uncertainties inherent in clinical trials and product development programs, the availability of funding to support continued research and studies, the availability or potential availability of alternative therapies or treatments, the availability of patent protection for the discoveries and strategic alliances, as well as additional factors that may cause Knopp's actual results to differ from our expectations. There can be no assurance that KNS-760704 will be successfully developed or manufactured or that final results of clinical studies will be supportive of regulatory approvals required to market the products. Knopp undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise

Tuesday, March 10, 2009

FDA, Insmed, Inc. Announce Special Program for IPLEX and ALSThe U.S. Food & Drug Administration granted approval for a limited number of people with ALS in the United States to receive IPLEX, a drug that combines insulin-like growth factor (IGF-1) and IGF binding protein 3. IPLEX, which has not been approved for use in ALS, is manufactured by Richmond, VA-based Insmed, Inc. The new program is the result of an agreement between the FDA and Insmed.The ALS Association hopes that the FDA-approved program will develop informative data about IPLEX that can lead to a better understanding of its efficacy and safety and enable both patients and clinicians to make more informed decisions about the use of IPLEX and its potential as a therapy for ALS. To this end, The Association encourages the FDA and Insmed to establish partnerships with the ALS community to ensure that the program yields meaningful results that will guide the next steps in determining whether IPLEX is effective and safe for ALS.IPLEX originally was approved in the United States as a treatment for children with growth failure, but it is now discontinued and no longer available for this population. The drug is being tested in a now-closed study involving myotonic muscular dystrophy (MMD). Insmed is supplying IPLEX to the Italian government under an “expanded access program,” but it continues to be an untested and unproven treatment for ALS in the United States.Based on existing clinical and scientific evidence, The ALS Association cannot encourage or recommend the off-label use of this medication without substantive evidence of its efficacy through a rigorous clinical trial. The ALS Association is continuing to monitor and assess information about IPLEX as it becomes available to provide the public with the most up-to-date reports about its potential for ALS.Additional information on today's announcement is available here: http://www.fda.gov/cder/drug/infopage/mecasermin_rinfabate/default.htm.For additional information from The ALS Association, please contact our Patient Services Department at alsinfo@alsa-national.org.

Monday, March 9, 2009

Obama overturns Bush policy on stem cellsBy PHILIP ELLIOTT, Associated Press Writer Philip Elliott, Associated Press Writer 26 mins agoWASHINGTON – Reversing Bush policy, President Barack Obama on Monday cleared the way for a significant increase in federal dollars for embryonic stem cell research and promised no scientific data will be "distorted or concealed to serve a political agenda."Obama signed the executive order on the divisive stem cell issue and a memo addressing what he called scientific integrity before an East Room audience packed with scientists. He laced his remarks with several jabs at the way science was handled by former President George W. Bush."Promoting science isn't just about providing resources, it is also about protecting free and open inquiry," Obama said. "It is about letting scientists like those here today do their jobs, free from manipulation or coercion, and listening to what they tell us, even when it's inconvenient especially when it's inconvenient. It is about ensuring that scientific data is never distorted or concealed to serve a political agenda and that we make scientific decisions based on facts, not ideology."He said his memorandum is meant to restore "scientific integrity to government decision-making." He called it the beginning of a process of ensuring his administration bases its decision on sound science; appoints scientific advisers based on their credentials, not their politics; and is honest about the science behind its decisions.Fulfilling a campaign promise, Obama signed the order that on stem cell research that supporters believe could uncover cures for serious ailments from diabetes to paralysis. Proponents from former first lady Nancy Reagan to the late actor Christopher Reeve had pushed for ending the restrictions on research.Obama paid tribute to Reeve, calling him a tireless advocate who was dedicated to raising awareness to the promise of research.Obama's action reverses Bush's stem cell policy by undoing his 2001 directive that banned federal funding for research into stem lines created after Aug. 9, 2001.The president said his administration would work aggressively to make up for the ground he said was lost due to Bush's decision, though it can't be known how much more federal money will be spent on the research until grants are applied for and issued."Medical miracles do not happen simply by accident," Obama declared.Embryonic stem cells are master cells that can morph into any cell of the body. Scientists hope to harness them so they can create replacement tissues to treat a variety of diseases — such as new insulin-producing cells for diabetics, cells that could help those with Parkinson's disease or maybe even Alzheimer's, or new nerve connections to restore movement after spinal injury.House Minority Leader John Boehner, R-Ohio, criticized Obama, saying in a statement that the president had "rolled back important protections for innocent life, further dividing our nation at a time when we need greater unity to tackle the challenges before us."Bush limited the use of taxpayer money to only the 21 stem cell lines that had been produced before his decision. He argued he was defending human life because days-old embryos — although typically from fertility clinics and already destined for destruction — are destroyed to create the stem cell lines.The Obama order reverses that without addressing a separate legislative ban, which precludes any federal money for the development of stem cell lines. The legislation, however, does not prevent funds for research on those lines created without federal funding.Researchers say the newer lines created with private money during the period of the Bush ban are healthier and better suited to creating treatment for diseases.Obama called his decision a "difficult and delicate balance," an understatement of the intense emotions generated on both sides of the long, contentious debate. He said he came down on the side of the majority of Americans who support increased federal funding for the research, both because strict oversight would prevent problems and because of the great and lifesaving potential it holds."Rather than furthering discovery, our government has forced what I believe is a false choice between sound science and moral values," Obama said. "In this case, I believe the two are not inconsistent. As a person of faith, I believe we are called to care for each other and work to ease human suffering."Obama warned against overstating the eventual benefits of the research, but he said his administration "will vigorously support scientists who pursue this research," taking another slap at Bush in the process."I cannot guarantee that we will find the treatments and cures we seek. No president can promise that. But I can promise that we will seek them actively, responsibly, and with the urgency required to make up for lost ground," he said.It's a matter of competitive advantage globally as well, the president argued."When government fails to make these investments, opportunities are missed. Promising avenues go unexplored," Obama said.But the president was insistent that his order would not open the door to human cloning."We will develop strict guidelines, which we will rigorously enforce, because we cannot ever tolerate misuse or abuse," Obama said. "And we will ensure that our government never opens the door to the use of cloning for human reproduction. It is dangerous, profoundly wrong, and has no place in our society, or any society."

Please review/double-click, the hyperlink above.It contains some interesting stuff, to say the least.Quick excerpt:"This is the first demonstration of transplanted human neurons synapsing, or making mature structural connections, with the rat motor neurons, something which has not been demonstrated before," said Dr. Karl Johe, Neuralstem's Chief Scientific Officer and a study co-author. "Our earlier work with this ALS model showed that the stem cells delayed onset of the disease and played a neuroprotective role. Now we have clear evidence that they can become an integral part of the rat nervous system that controls the muscles. I would expect these cells to be readily accepted by and integrated into a human nervous system, such as in an ALS or a spinal cord injury patient."

I believe you both saw this on ALSTDI forum, where I posted it March 2. Nothing has changed. The FDA will do what they’re going to do—with or without Italian data. This is no longer an issue of science (data about whether Iplex is or is not pre-proven and good for those with ALS). It’s simply an issue of whether or not they can deny a terminal population a medication that is already FDA approved—nothing else. That’s an issue of state statutes, federal law and FDA practice—all of which they (FDA) are now violating. And those physicians who will write off-label Rx’s or INDs will do so with or without Italian data—which they find suspect under the best of situations. And those who won’t write off-label or INDs will not start doing so because they see data from an Italian “observation.”

Anyone reading this can use their time more constructively by writing to your legislators and to FDA, using as a template that which you have previously seen from several people—me, Eddie Esparza and others. Or, write a new and different message demanding the right to use Iplex.

Regards,Steve Byer

From ALSTDI FORUM March 2, 2009

Dear Randy,When reading the following, please bear in mind that I am neither an apologist nor spokesperson for Insmed. Because of my interest in Iplex as a potential treatment for ALS, I have been able to make some communication inroads with Insmed and others related to this issue. I think, from your post quoted just below, and others from you more recently, it is advisable to share some thoughts on the subject.“I feel like a broken record and apologies if this has been covered in another post, but it has now been almost 3 months since the Iplex rally, at which time we were told (those of us who attended) that the Italian data would be released soon. I understood that it was this data that was to be the predicate of further activities and pursuits of obtaining it in the U.S. and maybe elsewhere. Have I missed something? Has this data been released or, if not, any indication as to when this will happen? I suppose "soon" could be anytime, but with each passing day an no news on this front, it looks more and more like either there was misinformation, miscommunication or attempts by one or more parties to mislead and spread false hope. I hope I'm wrong about this, of course.” Questioner ALS-TDI forum 02/09/09In response to the above and other of your posts and emails:1. When I asked for the data on December 22, Insmed stated they have turned over the results to two separate researchers for analysis and comparison to historical placebos. They had, just then, received the data from Italy after “numerous requests.”2. When I asked again on January 19, 2009, they stated both companies were having difficulty analyzing the information received from Italy because of the difficult nature of translating data from many different areas of Italy (20), many different age and progression groups, many different analyses by physicians treating the patients (I think it was 20 physicians) and few patients in the study (only 60 because they excluded all others who weren’t on Iplex for at least a certain time period). In other words, it was never purported to be a clinical test study—but rather “an observation.”3. While the information is unsatisfactory to me, and perhaps to you, it’s also understandable even by those of us who would prefer a clear set of study data and results from a well-conducted double-blind test study.4. Those with ALS don’t always have the luxury of time that a clinical test study requires. In the case of Myotrophin, an inferior drug for the purpose of treating ALS, it took from 1997 to 2009 for the results to be finalized. Even then, the results were unclear and marred by ineffective dosages and contradictory results from three different trials (in US, EU and again US).5. Therefore, those with diseases such as ALS (little-understood, no apparent cure, almost always treated unsuccessfully) are sometimes required to try a medication even without the benefit of prior test data if there is some, or any, reason to feel the treatment has promise. In the case of Iplex, there are several foundations, or at least suggestions, to feel the drug has promise. 6. You already know what those are, but just in case: prior usage by eight US patients in early 2007; continuing usage by now of up to 180 Italian patients paid for by the Italian government (a cost I doubt they take lightly); the results thus far of use by those with Myotonic muscular dystrophy (University of Rochester—a continuing study); scientific foundations previously provided you and others regarding the underlying benefits of IGF-1/IGFbp-3 vs free IGF-1; hope for Iplex when other hope doesn’t exist.7. Insmed has nonetheless stated that, from the little information on hand, there appears to have been a positive effect from at least 50% of those who were in the 60-patient group. That is my recollection of the specific words, and while it may be ever-so-slightly off, the meaning was clear—50% of those with ALS treated with anything is a success. I didn’t think to ask, at the time, if the remaining 50% were “stable, declining, or otherwise.”8. I was asked, at the time of the conversations above, not to publicize the information because it was unconfirmed and to some extent conjecture. Further, Insmed, as a public company, is unable to release such information without qualification because it can be construed as “forward-looking statements”, a legal issue for public companies. I do feel that enough time has passed from the conversations that it can be disseminated.9. If this is, in anyone’s view, “misinformation, miscommunication or attempts by one or more parties to mislead and spread false hope”, then so be it. Steve Byer

Sunday, March 8, 2009

Stem cells can help regrow diseased musclesSydney, March 6 : An experimental procedure that dramatically strengthens stem cells' ability to regenerate damaged tissue could offer new hope to victims of muscle-wasting diseases like myopathy and muscular dystrophy.The first-ever procedure has been successfully used to regrow muscles in a mouse model, but it could be applied to all tissue-based illnesses in humans in the liver, pancreas or brain, the researchers say. The research team, which is based at University of New South Wales (UNSW) and formerly from Sydney 's Westmead Children's Hospital, adapted a technique currently being tried in bone marrow transplantation. Adult stem cells are given a gene that makes them resistant to chemotherapy, which is used to clean out damaged cells and allow the new stem cells to take hold. The ability of adult stem cells to regenerate whole tissues opens up a world of new possibilities for many diseases, according to the study co-authors, Peter Gunning and Edna Hardeman, both professors, and Antonio Lee, from UNSW's School of Medical Sciences. "What has been the realm of science fiction is looking more and more like the medicine of the future," Gunning said. The procedure solves one of the major hurdles involving stem cell therapy - getting the cells to survive for more than an hour or so after inserting them into damaged tissue, said an UNSW release. These findings were detailed in the journal Stem Cells.

How stem cells turn into blood cellsWashington, Mar 6 : A research team led by an Indian origin scientist has shed light on how stem cells turn into blood cells.Stem cells are the building blocks of every organ and tissue in the body. They have a unique ability to become any type of cell in the body including bone, muscle and blood cells.Dr. Mick Bhatia, director of the McMaster University Stem Cell and Cancer Research Institute claim to have identified a particular cell pathway, known as the noncanonical Wnt that prompts stem cells to specialize and become blood cells.The pathway appears to organize the stem cells so that they can respond to signals telling them what to turn into."By directing cell differentiation, this method provides the most efficient way to produce blood cells that we are aware of to date," said Bhatia."This finding is exciting because it may provide a new way to make blood from human stem cells that could be used to regenerate the blood system in patients, including those with leukemia or those undergoing cancer treatments that indirectly destroy the immune and blood system," said Dr. Christine Williams, Director of Research Programs at the Canadian Cancer Society Research Institute.The findings are published in Cell Stem Cell.

Friday, March 6, 2009

Source: Obama to reverse limits on stem cell workBy BEN FELLER and LAURAN NEERGAARD, Associated Press Writers Ben Feller And Lauran Neergaard, Associated Press Writers 1 min agoWASHINGTON – President Barack Obama expected to sign an executive order on Monday reversing restrictions on federal funding of embryonic stem cell research.The long-expected move is likely to stir up not only the promise of scientific breakthrough but also the controversy over where government crosses a moral line.Obama will hold an event at the White House to announce the move, a senior administration official said Friday. The official spoke on condition of anonymity because the policy had not yet been publicly announced.Under President George W. Bush, federal money for research on human embryonic stems cells was limited to those stem cell lines that were created before Aug. 9, 2001. No federal dollars could be used on research with cell lines from embryos destroyed from that point forward.Obama's move is expected to lift that restriction. The official said the aim of the policy is restore "scientific integrity" to the process.Embryonic stem cells are master cells that can morph into any cell of the body. Scientists hope to harness them so they can create replacement tissues to treat a variety of diseases — such as new insulin-producing cells for diabetics or new nerve connections to restore movement after spinal injury."I feel vindicated after eight years of struggle, and I know it's going to energize my research team," said Dr. George Daley of the Harvard Stem Cell Institute and Children's Hospital of Boston, a leading stem cell researcher.Such research is controversial because embryos must be destroyed to obtain the cells; they typically are culled from fertility-clinic leftovers otherwise destined to be thrown away. Once a group of stem cells is culled, it can be kept alive and propagating in lab dishes for years.There are different types of stem cells, and critics say the nation should pursue alternatives to embryonic ones such as adult stem cells, or those found floating in amniotic fluid or the placenta. But leading researchers consider embryonic stem cells the most flexible, and thus most promising, form — and say that science, not politics, should ultimately judge."Science works best and patients are served best by having all the tools at our disposal," Daley said.Obama made it clear during the campaign he would overturn Bush's directive.During the campaign, Obama said, "I strongly support expanding research on stem cells. I believe that the restrictions that President Bush has placed on funding of human embryonic stem cell research have handcuffed our scientists and hindered our ability to compete with other nations."He said he would lift Bush's ban and "ensure that all research on stem cells is conducted ethically and with rigorous oversight.""Patients and people who've been patient advocates are going to be really happy," said Amy Comstock Rick of the Coalition for the Advancement of Medical Research.The ruling will bring one immediate change: As of Monday, scientists who've had to meticulously keep separate their federally funded research and their privately funded stem cell work — from buying separate microscopes to even setting up labs in different buildings — won't have that expensive hurdle anymore.Next, scientists can start applying for research grants from the National Institutes of Health. The NIH already has begun writing guidelines for what embryonic stem cell lines will qualify under Obama's ruling. Among other things, the guidelines are expected to demand that the cells were derived with proper informed consent from the woman or couple who donated the original embryo.

The 1.5-million-euro study, which looked at the so-called 'sporadic' type of ALS, identified a gene called Sunc1 which appears to play a predominant role in regulating ALS. But Chio' said Sunc1 was "probably just the tip of the iceberg." He said the study will now move into a third phase in which 300 new patients will be examined, all of them Italian.

People's Republic of China.Results of recent investigations have demonstrated the plasticity of mesenchymal stem cells (MSC) can differentiate into neural lineages. In this study, we explored the experimental condition of differentiation into neuron-like cells or rhodopsin (RHOS)-positive cells induced by epidermal growth factor (EGF) and taurine in vitro and to investigate their biological characteristics. MSC were obtained from umbilical cord blood (UCB) of term deliveries. Cultured cells were treated with Dulbecco's modified Eagle's medium/F12 (pH 7.0-7.2) supplemented with 30 ng/ml EGF. After the third cell passage, the cells were trysinized and analyzed with a flow cytometer using the following monocloned antibodies: CD90, CD29, CD34, CD44, and CD45. Taking another MSC of the third passage, its basal medium was replaced with alpha minimum essential medium supplemented with taurine (50 micromol/L). Cells were cultured for an additional 8-10 d, fixed, and then immunocytochemicall y analyzed. Primary antibodies included the following: neuron-specific enolase (NSE), RHOS, and nestin. In our study, we isolated a cell population derived from UCB, which possesses morphological characteristics similar to those of MSC isolated from bone marrow. In the cytometric analysis, MSC did not present labeling for the hematopoietic line (CD34 and CD45) and were positive for CD29, CD44, and CD90. After induction by taurine, 80.5 +/- 16.2% of the cell population expressed NSE, 36.8 +/- 9.6% expressed RHOS, and 29.6 +/- 9.3% expressed Nestin, while only 7.9 +/- 3.5% expressed NSE in the control group. This study demonstrates that partial MSC induced by taurine and EGF can differentiate into neuron-like cells or RHOS-positive cells in vitro, which may provide a promising therapeutic strategy for the treatment of some forms of retinal degeneration.

Monday, March 2, 2009

Stem cell breakthrough now goes one step furtherSun Mar 1, 1:20 pm ETPARIS (AFP) – Pioneering work by Japanese stem-cell researchers two years ago has taken a major step forward, helping the quest for versatile, grow-in-a-dish transplant tissue, according to papers published on Sunday.Two teams have combined ideas to devise a safer technique for reprogramming skin cells so that they become "pluripotent" stem cells -- fundamental cells that then grow into specialised organs.Their effort builds on an award-winning breakthrough in 2007 by Shinya Yamanaka of Kyoto University.He and his team introduced four genes into skin cells, reprogramming them so that they became indistinguishable from embryonic stem cells.That achievement conjured the distant vision of an almost limitless source of transplant material that would be free of controversy, as it would entail no cells derived from embryos.But the downside of the technique for creating these so-called induced pluripotent stem cells (iPS) is that the genes are delivered by a "Trojan horse" virus.Reprogramming cells using a virus modifies their DNA in such a way that they cannot be given to patients without boosting the risk of cancer.In the new studies, published by the British-based journal Nature, two squads of researchers from Britain and Canada recount a method by which the four genes are delivered into the cell without using a virus -- and then are removed after the reprogramming is done.The insertion is carried out using "piggyBac," a tried-and-tested technique in genetically-modified crops in which mobile genetic sequences called transposons are slotted into the genome.In the iPS work, it has been tested successfully on mouse and human skin cells. Tests on the reprogrammed cells lines show they faithfully reproduce the behaviour of embryonic stem cells."I was very excited when I found stem cell-like cells in my culture dishes. Nobody, including me, thought it was really possible," said Keisuke Kaji from the Centre for Regenerative Medicine at the University of Edinburgh, Scotland.In a press release issued by Britain's Medical Research Council, Ian Wilmut -- the "father" of Dolly the cloned sheep -- stressed that the new iPS cells would have to be tested thoroughly for safety before being used in any human trials.And, "crucially," scientists were still hunting for a way of coaxing pluripotent cells into the specialised tissues that could be used in transplants, said Wilmut, who heads the unit where Kaji works.Even so, "there is hope that the promise of regenerative medicine could soon be met," he said.Stem cells have excited huge interest over the past decade. Promoters say this material could reverse cancer, diabetes, Alzheimer's and other regenerative diseases.But the dynamic has been sapped by opposition from religious conservatives, who argue that research on embryos -- the prime source of stem cells so far -- destroys human life.

Saturday, February 28, 2009

Stem cell research supporters offer U.S. Senate billBy Maggie Fox Maggie Fox Fri Feb 27, 3:35 pm ETWASHINGTON (Reuters) – Two prominent supporters of stem cell research said on Thursday they had reintroduced a Senate bill that would allow federal funding for human embryonic stem cell research, in anticipation of President Barack Obama's support for the work.Senators Tom Harkin, an Iowa Democrat, and Arlen Specter, a Republican from Pennsylvania, said their bipartisan measure would allow federal funding for research using stem cells taken from human embryos left over from fertility treatments."It is the same bill that both houses of Congress approved in 2007, but was vetoed by President Bush," they said in a statement.Obama has promised to overturn Bush's policy that strictly limited the use of federal funds for embryonic stem cell research.Many groups that support embryonic stem cell research have been eagerly waiting for him to do so, but White House spokesman Robert Gibbs has hinted that Obama would prefer to wait and do something in concert with Congress."For too long, political interference has delayed research that holds the promise for millions of Americans who suffer from a wide range of diseases," Harkin said in a statement."President Obama has promised to lift the restrictions on embryonic stem cell research that were put in place by President Bush, and I hope and expect that he will do so soon, but we have to make sure that the freedom to pursue this research is also protected by federal law, not merely by an executive order that can be reversed during a future administration."Specter said legislation would protect the policy "so that it does not ping-pong back and forth with each successive president."Other senior senators co-sponsored the bill, including liberal Democrats Edward Kennedy of Massachusetts and Dianne Feinstein of California, as well as conservative Republican Orrin Hatch of Utah.Stem cells are the body's master cells and those taken from days-old embryos are especially powerful, with the ability to change into all the various cell-types in the body.Researchers are studying them to try and find ways to regenerate tissues and treat diseases such as Parkinson's, diabetes, cancer and also injuries.Opponents such as Bush say it is immoral to experiment on or destroy human embryos and say U.S. taxpayers should not have to pay for such research.

Thursday, February 26, 2009

Public release date: 23-Feb-2009[Print ArticleE-mail ArticleClose Window ]Contact: Donna Perrydonna@biologists.com44-122-343-3319The Company of BiologistsHuman stem cells provide a new model for Lou Gehrig's diseaseMotor neurons derived from embryonic stem cells mimic the progress of familial ALSFebruary 23, 2009, Cambridge, UK – Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a devastating condition in which motor neuron degeneration causes progressive loss of movement and muscle tone, leading to death. Overcoming the limited success of previous models, a report published in Disease Models & Mechanisms (DMM), dmm.biologists.org describes how neurons can be derived from human stem cells, and engineered to mimic inherited ALS.Researchers at the University of California Los Angeles developed an optimized protocol to generate motor neurons from human embryonic stem cells (ES cells), which express normal or mutant forms of the SOD-1 gene, which is linked to inherited, familial ALS. Resulting cells exhibit hallmark characteristics of motor nerve cells, and neurons expressing mutant SOD-1 display abnormalities typical of ALS. Defects included shortened cell projections and a reduced life span compared to cells containing the normal SOD-1 gene.This human cell-derived model of ALS provides a new method of studying this disease and testing novel therapeutics. This is especially helpful as only one drug is approved to help slow ALS progression, and animal models currently used in drug development have had limited success. Additionally, this research may aid other gene-linked neurodegenerative diseases, as they too may benefit from studies in a human cell-derived model.###Commentary on this work will be featured in the DMM Podcast for Volume 2, Issue 3/4 of DMM. Podcasts are available via the DMM website at: dmm.biologists.org.The report was written by Saravanan Karumbayaram, Theresa K. Kelly, Andres A. Paucar, Anne J.T. Roe, Joy A. Umbach, Andrew Charles, Harley I. Kornblum, and Martina Wiedau-Pazos at the David Geffen School of Medicine at the University of California Los Angeles, and Steven A. Goldman at the University of Rochester Medical Center in Rochester, NY. The report is published in the March/April issue of Disease Models & Mechanisms (DMM), a research journal published by The Company of Biologists, a non-profit based in Cambridge, UK.About Disease Models & Mechanisms:Disease Models & Mechanisms (DMM) is a new research journal publishing both primary scientific research, as well as review articles, editorials, and research highlights. The journal's mission is to provide a forum for clinicians and scientists to discuss basic science and clinical research related to human disease, disease detection and novel therapies. DMM is published by the Company of Biologists, a non-profit organization based in Cambridge, UK. The Company also publishes the international biology research journals Development, Journal of Cell Science, and The Journal of Experimental Biology. In addition to financing these journals, the Company provides grants to scientific societies and supports other activities including travelling fellowships for junior scientists, workshops and conferences. The world's poorest nations receive free and unrestricted access to the Company's journals.

In one of the most significant breakthroughs in the recent history of ALS research, a consortium of scientists organized and funded by The ALS Association has discovered a new gene, ALS6 (Fused in Sarcoma), responsible for about 5 percent of the cases of inherited ALS. The discovery will provide important clues to the causes of inherited ALS, which accounts for 10 percent of all cases, and sporadic ALS, which occurs in individuals with no family history of the disease and accounts for the other 90 percent of cases diagnosed.“This is a momentous discovery in furthering our understanding of ALS,” said Lucie Bruijn, Ph.D., senior vice president of Research and Development at The ALS Association. “A new gene provides a new piece of the puzzle we can use to shed light on why ALS develops, and where to focus our efforts on creating new treatments and finding a cure.”The results of this groundbreaking research are published in the Friday, February 27 issue of the prestigious journal Science. The project was led by Tom Kwiatkowski M.D., Ph.D., at Massachusetts General Hospital, and Robert Brown, M.D., of the University of Massachusetts School of Medicine, and ALS Association-funded researchers Caroline Vance, Ph.D., and Christopher Shaw, M.D., of Kings College in London. The project was supported by a consortium of leading ALS researchers from around the world, formed as part of The Association’s Gene Identification Project. Their success reflects an unprecedented effort to accelerate the search for genetic mutations linked to all forms of ALS.Dr. Brown noted, “We are particularly delighted because our trans-Atlantic consortium has pursued the chromosome 16 gene for more than six years. The ALS Association has been an all-important partner in this search. This discovery should lead to new cell and animal models of ALS, which will accelerate drug development.”“Global partnerships between investigators and funding agencies, such as the Motor Neuron Disease Association in the United Kingdom, are crucial to making these kinds of breakthroughs,” Dr. Bruijn commented. “This finding has opened up a whole new avenue of research and has the potential to uncover a common mechanism for most forms of ALS.”The gene mutations were first identified by Dr. Kwiatkowski and were immediately confirmed by Dr. Vance, who also demonstrated abnormal accumulations of the mutant protein in cells cultured in the laboratory and the motor neurons of people carrying FUS mutations.The gene, called FUS (“fused in sarcoma”), normally carries out multiple functions within motor neurons. These include regulating how gene messages (called messenger RNAs) are created, modified, and transported in order to build proteins. Some of these same functions also are performed by another gene called TARDPB encoding the protein TDP43, and mutations in the TDP-43 gene were recently linked to ALS as well.“The fact that these two genes help perform the same function suggests that problems in this function may be critical in the development of ALS,” Dr. Bruijn said. “More research into exactly how these two genes work could ultimately lead to new treatments that are effective in slowing or stopping the progression of ALS and extending the lives of people with the disease.”The mutations in the ALS6 gene were identified by detailed genetic sequencing in several families with an inherited form of ALS (familial ALS). Normally, the ALS6 protein works in the cell’s nucleus, but the mutations caused it to instead cluster outside the nucleus. Further work will be needed to determine precisely how this leads to ALS. With the gene in hand, scientists will be able to create cell and animal models containing the mutated gene, to examine in detail how the mutation operates and how it causes ALS.“This suggests there may be a common mechanism underlying motor neuron degeneration,” according to Dr. Shaw. Motor neurons are nerve cells in the brain and spinal cord that control muscles. Motor neurons degenerate in ALS.This is the second ALS-causing gene to be discovered in the past 12 months. SOD1, discovered in 1993, accounts for 20 percent of inherited cases of the disease. Mutations in the TARDP gene account for another four to five percent. The only well-defined causes of ALS are genetic. In both inherited and sporadic ALS, the disease symptoms and pathology are the same.The possibility that ALS may be caused by several factors is the rationale for The Association’s policy of funding multiple genetic projects around the world and encouraging these leading geneticists to work together and share information to help locate disease-linked genes for faster, more accurate scientific results. By funding research on a global level, The Association helps put together “genetic pieces” of the ALS puzzle.“Through our support of research such as this study, The ALS Association is committed to finding the causes of ALS, and using that knowledge to develop a cure as rapidly as possible,” Dr. Bruijn said. “We will build on the discovery of this new gene to carry that effort forward.”

Wednesday, February 25, 2009

I am once again, requesting help from any and all family, friends, & supporters . Please Review the above attachments we (Team IPLEX) have provided...

You can... cut and paste certain talking points from the attachments, or... you can just cut and paste the entire document - as is. (of course don't forget to, change the address, name, and salutation's.) then double-click each Web link below, (which will open up that particular FDA officer's e-mail voicing your support for this (PALS) cause.

All We Are Asking ... is that they (the FDA hierarchy), provide us (PALS) a more data-driven reason/argument, for denying American PALS a drug/therapeutic, that the Italian government are paying Millions of Dollars annually (on behalf of their citizenry), for the specific Treatment of ALS. Is That Really Too Much to Ask ?... Just show us the proof, that -- IPLEX Is Dangerous, and we'll all go away and shut up.

What's with all the secrecy ? What ever happened to openness and transparency? - What's with the... MY WAY OR HIGHWAY attitude of Dr. Katz? ... Finally, why haven't the Italian government, refused to stop paying, the Millions of Dollars (annually), for reasons that, IPLEX Is Dangerous and or Ineffective.?

Yes, roadblocks (FDA ) exist that prevent immediate access to Iplex ,but just like the last time we went up against Goliath, we are making progress. Last week, Stephen Byer (Team IPLEX Capt.) & his nonprofit organization - ALS worldwide, met with two Illinois legislators, who have promised, and are already showing, their support, of our effort to secure Iplex.

Therefore, we (Team IPLEX) are attaching documents in both PDF format and Microsoft Word format for your immediate use:

Please Take 10-15 minutes and Help, because for myself & other PALS, it truly is - A Race against Time.

Either of these documents can be copied and pasted into either media or congressional websites by using the Microsoft Word versions attached. Or, they can be sent, as is, to those for whom you have an email address by using the PDF files as attachments.

At the latter part of 2008, we (Team IPLEX & supporters) were collectively successful in doing what some said, was impossible - we can do it again but not without your help.

Immediately below are FDA contacts beginning with Dr. Russell Katz, the individual who appears responsible for the current FDA decision to deny Iplex to the ALS community. We urge that you send a copy of both the Congressional letter and the press release to each and every one of the following FDA personnel.

Friday, February 20, 2009

NeuralStem Inc.'s ALS Trial on Clinical Hold 2/20/2009ROCKVILLE, Md., Feb. 20 /PRNewswire-FirstCall/ -- Neuralstem, Inc. announced today its spinal cord stem cell trial to treat ALS is on clinical hold and that the Federal Drug Administration (FDA) has provided the Company with specific comments, questions and recommendations for modifications to its protocol.(Logo: http://www.newscom.com/cgi-bin/prnh/20061221/DCTH007LOGO )"The FDA has presented us with their review of our entire Investigational New Drug (IND) application," said Richard Garr, Neuralstem's President & CEO. "They have asked for some additional information regarding our product manufacturing process and pre-clinical studies, as well as our novel clinical delivery injection device and technique. The Company believes that it can provide this information in an expeditious manner.""The Agency has also requested various modifications to the protocol and eligibility criteria for patients in the trial, as well as slight changes to the timing of the surgeries," Mr. Garr continued. "We are evaluating these changes and will respond accordingly. The Agency had extensive 'non hold' comments, requests for information, and recommendations. These primarily concerned issues that will need to be addressed for final product manufacturing and testing. We are appreciative of their work in this area.""Over all we believe the Agency's comments and recommendations are extremely helpful," Garr concluded. "We are evaluating them carefully, and expect to reach agreement with the Agency on all matters so that the trial can be approved and move forward."About NeuralstemNeuralstem's patented technology enables, for the first time, the ability to produce neural stem cells of the human brain and spinal cord in commercial quantities, and the ability to control the differentiation of these cells into mature, physiologically relevant human neurons and glia. Major Central Nervous System diseases targeted by the Company with research programs currently underway include: Ischemic Spastic Paraplegia, Traumatic Spinal Cord Injury, Huntington's disease and Amyotrophic Lateral Sclerosis (ALS).In pre-clinical work, the company's cells have extended the life of rats with ALS (Lou Gehrig's disease) as reported the journal TRANSPLANTATION, in collaboration with Johns Hopkins University researchers, and also reversed paralysis in rats with Ischemic Spastic Paraplegia, as reported in NEUROSCIENCE on June 29, 2007, in collaboration with researchers at University of California San Diego.Cautionary Statement Regarding Forward Looking InformationThis news release may contain forward-looking statements made pursuant to the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Investors are cautioned that such forward-looking statements in this press release regarding potential applications of Neuralstem's technologies constitute forward-looking statements that involve risks and uncertainties, including, without limitation, risks inherent in the development and commercialization of potential products, uncertainty of clinical trial results or regulatory approvals or clearances, need for future capital, dependence upon collaborators and maintenance of our intellectual property rights. Actual results may differ materially from the results anticipated in these forward- looking statements. Additional information on potential factors that could affect our results and other risks and uncertainties are detailed from time to time in Neuralstem's periodic reports, including the annual report on Form 10- KSB for the year ended December 31, 2007 and the quarterly report on form 10-Q for the period ended September 30, 2008.CONTACT: Richard Garr, President of Neuralstem, Inc., +1- 301-366-4960; orMedia: Deanne Eagle of Planet Communications, +1-917-837-5866; orInvestors: Ina McGuinness of ICR, Inc., +1- 310-954-1100Read at BioSpace.com

Some thoughts on Geron's start of stem cell-based therapy for spinal injury.http://www.alscenter.org/news/briefs/090218.cfm Late last month, ALS sufferers' hearts had to quicken at the announcement by=the biotech firm, Geron, hailing the start of clinical trials of a stem cel=-based therapy for new spinal injuries. Though the trial is a Phase I study meant to see if injected cells are safe =or humans, and though its patient subjects have had their very specific spi=al cord injuries only one-to-two weeks, the appearance of an authentic tria= involving potentially therapeutic cells - ones aimed at lessening or preve=ting paralysis - is exciting.What about trying out those cells or similar ones for ALS injuries to the ne=vous system?"Packard researchers in this country and Europe are doing an enormous amount=of work to sort out the many steps necessary to discover which cells to use=one day in ALS patients," says Packard Director Jeffrey Rothstein. "But eve= after that most essential work, after the appropriate cell type stands wel= out from the others, questions remain. "These are not simply safety questions," Rothstein says: Many of our ALS pat=ents would be willing to try a new therapy and fully understand the risks.What needs answering are questions of benefit and of procedure. "How do you =dminister the cells," he asks, "so the risk of getting them is justified by=their potential to relieve the disease or improve quality of life? "The impediments to our progress now are typical of what you'd encounter for=any potential new therapeutic drug," he explains. How many cells are approp=iate to deliver? That's the equivalent of finding the appropriate dose of a drug.What's the best way to distribute the cells throughout the brain and spinal =ord? That is akin to asking how often you give a drug.Do you give the cells through an IV or by direct surgical injection? What ha=pens to cells after we put them in? Do they divide like tumors? Do they die=and cause a scar that would hasten ALS effects? "Those are questions we mu=t first sort out in animals," says Rothstein.And a cell therapy carries its own unique concerns: How do we know that the =good' cells we put into one patient are identical to those we'd culture and=give a patient a year later? Quality assurance issues are well-established =or drugs like penicillin, Rothstein adds, but they have to get worked out f=r every new type of stem cell. The Packard Center has been doing its homework, however - not just for its o=n scientists but for the world's.A DECADE OF GROUNDWORK For almost 10 years, our researchers have been laying the groundwork for pos=ible cell therapy for ALS. Many of their published studies have likely infl=enced the Geron trial-planners. Here are a few:- Packard scientist Doug Kerr was the first to show that an animal model of = paralytic disease could be helped by injections of stem cells. - Packard Director Jeff Rothstein and his team solidified the suspicion that=motor neurons' natural protectors are its neighboring glial cells - that th=y remove harmful toxins as they collect in nervous system tissues under ass=ult. (The cells injected in the Geron study have the potential to become gl=al cells, those widespread nervous system cells in close proximity to motor=neurons.) - Work by Don Cleveland and Jean-Pierre Julien sent researchers scurrying mo=e surely to explore the concept of glial cells as protectors. They revealed=that keeping glia healthy can protect even motor neurons carrying an ALS ge=e. - Nicholas Maragakis and Mahendra Rao showed investigators everywhere how to=isolate, culture and precisely identify glial-restricted precursor (GRP) ce=ls - the broad class of "undeveloped" cells akin to the cell types Geron is=injecting. - Maragakis and Rao demonstrated that GRPs are neuroprotective - both in cul=ures that mimic the nervous system and in live animal models of ALS. Maraga=is and Rothstein have gone on to clarify much of the underlying biology. MORE RECENT WORK Because they specialize in ALS biology, Packard scientists know to target st=m cell and related therapies where they'd potentially do the most good. Las= November, for example, they reported a key step, an animal study in which =hey injected glial-restricted precursors precisely around the cervical spin=l cord home to motor neurons that permit breathing. Paralysis of those neur=ns precipitates death in ALS. The rat ALS models in the study survived significantly longer. Their forelim=s and breathing muscles stayed strong and functional far longer, while moto= neuron loss slowed. "This shows us that targeting cell delivery to critica= spinal areas is certainly worthy of investigating as a therapy for ALS, as=a way to slow specific types of motor neuron loss," says researcher Nichola= Maragakis. Now work needed to inch closer to clinical trials is underway.* * *In the last six months, Maragakis and colleague Hongjun Song have been worki=g at what can only be described as ALS's leading edge: they've begun studyi=g human pluripotent stem cells created from the skin of a patient with fami=ial ALS. One of the few recent, legitimate scientific breakthroughs, the technique in=olves reprogramming the patient's skin cells to behave like stem cells. The= the stem cells are coaxed to become motor neurons. This means that Packard=scientists will be able to study the progress of real ALS disease in real h=man cells, something that was out of reach before. ___________________________________ About The Robert Packard Center for ALS Research at Johns Hopkinswww.alscenter.org Located in Baltimore, the Robert Packard Center for ALS Research at Johns Ho=kins is a worldwide collaboration of scientists aimed at developing therapi=s and a cure for amyotrophic lateral sclerosis (ALS), also known as Lou Geh=ig's disease.The Center is the only institution of its kind dedicated solely to the disea=e. Its research is meant to translate rapidly from the lab bench to the cli=ic, largely by eliminating time spent waiting for grants and lowering insti=utional barriers to sharing scientific results.Scientists and clinician members of the Packard Center have moved drugs reli=bly and rapidly from preclinical experiments to human trials. Direct or ind=rect links to international biotech or pharmaceutical companies bring the i=frastructure and experience needed to make promising drugs into therapies.Packard scientists are the first to propose and test a combination approach =o drug therapy, a tactic that has worked for AIDS, cancer and other disease=.ALS is a progressive, disabling neuromuscular disease that causes complete p=ralysis and loss of function - including the ability to eat, speak and brea=he. ALS progresses quickly and is not curable. Most patients die within fiv= years of diagnosis._________________________________________Rebecca BergerResearch Program CoordinatorRobert Packard Center for ALS Research at Johns Hopkins5801 Smith Avenue McAuley Suite 110Baltimore, MD 21209410.735.7678 direct410.735.7680 faxrberger6@jhmi.eduwww.alscenter.orgwww.fiesta5K.org****************************************************************************=***Click on the link below to unsubscribemailto:sympa@lists.johnshopkins.edu?subject=unsubscribe%20alscenter&body=3DPLEASE%20SEND%20THIS%20EMAIL%20OUT%20FOR%20UNSUBSCRIBINGPlease follow the instructions below if the above link does not work:Compose an email to: sympa@lists.johnshopkins.eduWith Subject: unsubscribe alscenterNote: 'unsubscribe alscenter' should be on the SUBJECT line, NOT IN THE MESS=GE BODY.To contact the list owner: mailto:alscenter-request@lists.johnshopkins.edu

Tuesday, February 17, 2009

We ( Team IPLEX ) are respectful requesting help from any and all family, friends, & supporters . Please Review attachments I have provided You can... cut and paste certain talking points from the attachments , or you can just cut and paste the entire document -- as is... of course,change the address , name, and salutation's. -- Then utilize the Web link that I have provided, to send your elected representatives (2) senators & ( 1) Congressperson a quick e-mail voicing your support for this (PALS) cause. All we are asking is that they (our elected representatives) look into the shady nature of this matter -- end of story... is that too much to ask?... just looked into the matter, on behalf of terminally ill people? -- Please Take 10-15 minutes and Help, because for PALS, it truly is -- A Race against Time. http://www.visi.com/juan/congress/http://www.senate.gov/general/contact_information/senators_cfm.cfm

The whole Iplex story significantly highlights the lack of care and concern by institutes such as the FDA. The human factor has been totally removed. The FDA's response has been unbelievable & shameful. They (FDA) are in effect shutting patients out of a drug/therapeutic , that they themselves have already (2006) approved for infants/children. Now, they (FDA) are claiming that it is necessary to shut/turn down all IND applications for IPLEX for reasons that it -- kills people? And get this, all based on unsubstantiated data. A Drug Well-Tolerated by Babies? In plain speak, there is something very fishy going on here. We, (Team IPLEX & supporters) feel that the FDA should be making a more concise data-driven argument, or at least before they start making politically motivated & bogus claims that IPLEX kills people... IPLEX has been supplied to Italian, PALS for over 2 years now, over 100 patients, how many of the 100 have died? Faster by using IPLEX.

Stephen & Barbara Byer parents of Ben Byer (famed, movie producer of the award-winning film "Indestructible" ) explain how their son, Ben, after 14 days of taking the drug (IPLEX ), went from "oatmeal and pudding to a Whoppers ." But was subsequently forced to stop taking the drug (IPLEX) when it was pulled/forced off the market by the terms of a patent lawsuit. We (team IPLEX & supporters) fought too hard in 2008, to get the companies to come to terms, (to make IPLEX available ) to shut up and go away now... .

Bottom line, WHAT DO WE (PALS ) HAVE TO LOSE? -- If our (PALS & supporters) current letter writing campaign, is able to inspire, motivate, and or raise awareness to the plight of PALS. I will definitely feel like --Mission Accomplished!! Even if we aren't able to get the FDA to immediately change, there bogus position, I still feel that victory is just around the corner, this fight will not be over... not by a long shot!! -- unless we give up and accept our fate.

If whatever we're able to accomplish with our current e-mail/ letter writing campaign helps the next generation of PALS dream bigger, work harder and once and for all, finish the work we've started, I'll always believed in my heart that we have made our mark as true champions. But Don't Get Me Wrong, I Want to Win This Thing ASAP... If It's Wrong and or Selfish to Want to Live Longer, well then,... I'm Wrong, & Selfish -- End of Story. PS: We Really Need Your Help

Sunday, February 15, 2009

Obama to lift ban on stem cell research soon: aideSun Feb 15, 10:01 am ETWASHINGTON (Reuters) – U.S. President Barack Obama will soon issue an executive order lifting an eight-year ban embryonic stem cell research imposed by his predecessor, President George W. Bush, a senior adviser said on Sunday."We're going to be doing something on that soon, I think. The president is considering that right now," Obama adviser David Axelrod said on "Fox News Sunday."In 2001, Bush limited federal funding for stem cell research only to human embryonic stem cell lines that already existed. It was a gesture to his conservative Christian supporters who regard embryonic stem cell research as destroying potential life, because the cells must be extracted from human embryos.Embryonic stem cells are the most basic human cells which can develop into any type of cell in the body.Scientists believe the research could eventually produce cures for a variety of diseases, including Parkinson's disease, diabetes, heart disease and spinal cord injuries.Obama vowed to reverse Bush's ban during his presidential campaign and in his inaugural address last month promised to return science to its proper place in the United States.The U.S. Food and Drug Administration last month cleared the way for the first trial to see if human embryonic stem cells could treat people safely.The trial will try to use stem cells from already existing lines to regrow nerve tissue in patients with crushed spinal cords.Stem cells are the body's master cells, giving rise to all the tissues, organs and blood. Embryonic stem cells are considered the most powerful kinds of stem cells, as they have the potential to give rise to any type of tissue.(Reporting by Alan Elsner; Editing by Eric Beech)Sun Feb 15, 10:01 am ETWASHINGTON (Reuters) – U.S. President Barack Obama will soon issue an executive order lifting an eight-year ban embryonic stem cell research imposed by his predecessor, President George W. Bush, a senior adviser said on Sunday."We're going to be doing something on that soon, I think. The president is considering that right now," Obama adviser David Axelrod said on "Fox News Sunday."In 2001, Bush limited federal funding for stem cell research only to human embryonic stem cell lines that already existed. It was a gesture to his conservative Christian supporters who regard embryonic stem cell research as destroying potential life, because the cells must be extracted from human embryos.Embryonic stem cells are the most basic human cells which can develop into any type of cell in the body.Scientists believe the research could eventually produce cures for a variety of diseases, including Parkinson's disease, diabetes, heart disease and spinal cord injuries.Obama vowed to reverse Bush's ban during his presidential campaign and in his inaugural address last month promised to return science to its proper place in the United States.The U.S. Food and Drug Administration last month cleared the way for the first trial to see if human embryonic stem cells could treat people safely.The trial will try to use stem cells from already existing lines to regrow nerve tissue in patients with crushed spinal cords.Stem cells are the body's master cells, giving rise to all the tissues, organs and blood. Embryonic stem cells are considered the most powerful kinds of stem cells, as they have the potential to give rise to any type of tissue.(Reporting by Alan Elsner; Editing by Eric Beech)

Saturday, February 14, 2009

Insmed Sells Follow-on Biologics Platform to Merck & Co., Inc. for Gross Proceeds of $130 Million--Insmed to Retain IPLEX(TM) portfolio Company to host conference call at 8:30 am ET todayRICHMOND, Va., Feb 12, 2009 /PRNewswire-FirstCall via COMTEX News Network/ -- Insmed Inc. (Nasdaq: INSM), a developer of follow-on biologics and biopharmaceuticals focused on niche markets with unmet medical needs, announced today that it has entered into a definitive agreement with Merck & Co., Inc. whereby Merck, through an affiliate, will purchase all assets related to Insmed's follow-on biologics platform. Under the terms of the agreement, Insmed will receive a total of $130 million for the assets. After fees, taxes and other costs related to the transaction, Insmed expects net proceeds of approximately $123 million as a result of this agreement.As part of this transaction, Insmed will receive initial payments of up to $10 million for Insmed's lead follow-on-biologic candidates and the remaining balance upon closing of the transaction, which is targeted for March 31, 2009. These initial payments will allow the Company to maintain its normal business operations throughout the closing period without the need for dilutive financing.Insmed's follow-on biologics assets include INS-19 and INS-20, whose development and commercial rights will now belong to Merck, as well as the Boulder, Colorado-based manufacturing facility. Merck intends to assume the facility's lease and ownership of all the equipment in the building. In addition, upon closing of the transaction, Merck intends to offer positions to employees of the Boulder facility. Insmed will retain its Richmond, VA corporate office, which houses its Clinical, Regulatory, Finance, and Administrative functions, in support of the continuing IPLEX(TM) program."We have long maintained that our follow-on biologics assets hold substantial value, and this agreement with Merck, one of the largest pharmaceutical companies in the world, is a testament to that value," said Dr. Geoffrey Allan, President and CEO of Insmed. "We are pleased that over the past two years our team has been successful in developing such a valuable asset, which, as a result of this agreement, will generate a substantial return to the Company.""This transaction will transform and strengthen our balance sheet in a completely non-dilutive fashion, and provides us with substantial financial flexibility in a market where cash, especially for small biotech companies, is scarce," continued Dr. Allan.The proceeds from the transaction will be used to support the continued development of IPLEX(TM), and the Company will carefully evaluate other options, which could include the distribution of a portion of the cash to shareholders.As of December 31, 2008, Insmed had $2.4 million in cash on hand with an ongoing net cash burn of approximately $1.2 million per month. This transaction, in accordance with Virginia corporate law, does not require a shareholder vote, though it is conditional on certain closing requirements, including obtaining necessary consents.RBC Capital Markets served as exclusive financial advisor to Insmed on the transaction and the review of strategic alternatives, and provided a fairness opinion to the Company's Board of Directors.Conference CallInsmed will host a conference call today at 8:30 AM ET in order to further discuss this transaction. To participate in today's 8:30 AM ET live conference call, please dial 800-891-3155 (U.S. callers) or 617-597-5527 (international callers), and provide passcode 54149478. A live webcast of the call will also be available at: http://phx.corporate-ir.net/playerlink.zhtml?c=122332&s=wm&e=2097821Please allow extra time prior to the webcast to register, download and install any necessary audio software.The webcast will be archived for 30 days, and a telephone replay of the call will be available for seven days beginning today at 12:30 PM ET at 888- 286-8010 (U.S. callers) or 617-801-6888 (international callers), using passcode 91512481.About InsmedInsmed Inc. is a biopharmaceutical company with unique protein process development and manufacturing experience and a proprietary protein platform aimed at niche markets with unmet medical needs. For more information, please visit http://www.insmed.com.Forward-Looking StatementsThis release contains forward-looking statements which are made pursuant to provisions of Section 21E of the Securities Exchange Act of 1934. Investors are cautioned that such statements in this release, including statements relating to planned clinical study design, regulatory and business strategies, plans and objectives of management and growth opportunities for existing or proposed products, constitute forward-looking statements which involve risks and uncertainties that could cause actual results to differ materially from those anticipated by the forward-looking statements. The risks and uncertainties include, without limitation, risks that closing conditions under our agreement with Merck & Co., Inc. may not be met, product candidates may fail in the clinic or may not be successfully marketed or manufactured, we may lack financial resources to complete development of product candidates, the FDA may interpret the results of studies differently than us, competing products may be more successful, demand for new pharmaceutical products may decrease, the biopharmaceutical industry may experience negative market trends, our continuing efforts to develop IPLEX(TM) may be unsuccessful, our common stock could be delisted from the Nasdaq Capital Market and other risks and challenges detailed in our filings with the U.S. Securities and Exchange Commission, including our Annual Report on Form 10-K for the year ended December 31, 2007. Readers are cautioned not to place undue reliance on any forward-looking statements which speak only as of the date of this release. We undertake no obligation to publicly release the results of any revisions to these forward-looking statements that may be made to reflect events or circumstances that occur after the date of this release or to reflect the occurrence of unanticipated events. Investor Relations Contact:Brian Ritchie - FD212-850-5683brian.ritchie@fd.comMedia Contact:Irma Gomez-Dib - FD212-850-5761Irma.gomez-dib@fd.com

MS (multiple sclerosis ) seems to offer a reasonable standard of comparison as it has about the roughly similar incidence as ALS (10,000 new cases per year for MS vs.7000 for ALS). There are approximately 400,000 people in the US living with MS vs. about 30,000 with ALS. Why the difference in prevalence if the diseases have a somewhat similar incidence rate? The major factor is lethality. MS patients live a normal life span while, ALS patients have a much shorter life span. If ALS were not such an effective killer, its prevalence rate would obviously be much higher. It seems reasonable that the two diseases should have nearly equal public funding, since they both occur with the similar frequency, but this is not the case. NIH budgets 169 million on MS research but $43 million on ALS? . It is immoral to base funding on prevalence in the context of similar incidence rates, thereby penalizing ALS patients for the lethality of their disease

The next disease for comparison is Huntington’s disease, another disorder caused by degeneration of brain cells, i.e. neurons, in certain areas of the brain. Its incidence and prevalence rate are much lower than ALS and patients usually live 10-25 years after diagnosis. Yet, this disease has an NIH budget of $51 million versus, $43 million for ALS.For the, 3rd and final comparison, HIV/AIDS. This illness has a large incidence (85,000) and prevalence (1,100,000) The number of patients who die from this disease each year is only a bit more than twice the number of ALS deaths, yet HIV/AIDS has a research budget of nearly $3 BILLION! vs. $41m for ALS-?

CONCLUSION I: Given its incidence rates and high lethality, ALS is woefully under-funded compared to other diseases or and this may explain, at least in part, why so little has been learned about this disease since Lou Gehrig’s death 65 years ago and why there have been no significant advances in treatment. It does not get the recognition it deserves because its high lethality severely limits the number of Americans who are living with the disease at any one point in time.

RECOMMENDATION I: The NIH budget for ALS research should be immediately increased to $110m or the same amount as MS.

The fact that the low prevalence rate of ALS, is directly due to its high lethality, therefore does not provide any incentive for major pharmaceutical companies to search for a cure. The only drug for ALS, Rilutek, about 15 years old now, extends life only 2-3 months, is used by many patients at high cost, yet the maker of the drug, Aventis, claims that it loses substantial money on Rilutek. MS, in comparison, with its 400,000 prevalence in the US alone, presents significant incentive for drug companies to invest in research. In fact, there are 5 meds that have proven very beneficial and sales are big. Avonex, for example, had close to $1b in sales in the last year. Another drug, Copaxone, may have the greatest potential to alter the course of MS and sales, once the issue of side effects is resolved, should be huge. If we accept the view that it costs the companies $800 m over 10 years to develop and bring to market a successful drug, then about $4 billion has been spent to produce these disease mitigating medications, or $400 m per year, not including current research.The FDA offers an orphan disease program to provide incentives to companies to invest in research of diseases having a prevalence of fewer than 200,000. The program provides tax incentives and other advantages for only small grants; unfortunately the incentives offered are too small to interest large companies with all their research potential.

Conclusion II: Because of ALS’s high lethality, prevalence will always be low. As a consequence, big pharma will not have the incentive to invest in ALS research and the high tech power of drug companies won’t be harnessed to find new therapeutic agents in the fight against ALS.

Recommendation II: Federal health agencies must provide the incentive to big pharma to invest in ALS research. The FDA Orphan Disease Program must be substantially increased to accomplish this. $80m per year in incentives must be allotted to get major companies interested in working on this research. Funding could include a combination of grants, tax credits and exclusive marketing rights, etc. The grants could be reduced as companies come closer to marketing the new agents.

Under-funding of ALS will not be righted without our ( PALS ) active intervention. I am asking for your help on behalf of both the 35,000+ Americans living with the disease today and the estimated 70,000 that ill die every 10 years , and the 7000 that will die just this year, until a cure is found. ALS has been neglected far too long.Sincerely,Edward W. Esparza(pals since 2005)"For PALS, the cost of waiting/doing nothing, is simply too high."

Wednesday, February 11, 2009

Dear Brothers and Sisters,

We ( Team IPLEX ) are respectful requesting help from any and all family, friends, & supporters . Please Review attachments I have provided

You can... cut and paste certain talking points from the attachments , or you can just cut and paste the entire document -- as is... of course,change the address , name, and salutation's. -- Then utilize the Web link that I have provided, to send your elected representatives (2) senators & ( 1) Congressperson a quick e-mail voicing your support for this (PALS) cause. All we are asking is that they elected representatives) looked into the shady nature of this matter -- end of story... is that too much to ask?... just looked into the matter, on behalf of terminally ill people? --

Please Take 10-15 minutes and Help, because for PALS, it truly is -- A Race against Time.

The whole Iplex story significantly highlights the lack of care and concern by institutes such as the FDA. The human factor has been totally removed. The FDA's response has been unbelievable & shameful. They (FDA) are in effect shutting patients out of a drug/therapeutic , that they themselves have already (2006) approved for infants/children. Now, they (FDA) are claiming that it is necessary to shut/turn down all IND applications for IPLEX for reasons that it -- kills people? And get this, all based on unsubstantiated data. A Drug Well-Tolerated by Babies?

In plain speak, there is something very fishy going on here. The company (INSMED) that developed & owns the property rights, refuses to release any data, from a 100 patient, 2 year trial/Italian program. We, (team IPLEX & supporters) feel that the FDA should be forcing this information out, in order to make a more concise data-driven argument, or at least before they start making claims that IPLEX kills people... Insmed has been supplying Iplex to Italian, PALS for over 2 years now to over 100 patients, respectively review this is Insmed has issued many, a statement that IPLEX is safe and well tolerated. IPLEX is already an FDA approved drug and has already been proven safe for an indication for infants/kids dwarfism ).

Stephen & Barbara Byer parents of Ben Byer (famed, movie producer of the award-winning film "Indestructible" ) explain how their son, Ben, after 14 days of taking the drug (IPLEX ), went from "oatmeal and pudding to a Whoppers ." But was subsequently forced to stop taking the drug (IPLEX) when it was pulled/forced off the market by the terms of a patent lawsuit. We (team IPLEX & supporters) fought too hard in 2008, to get the companies to come to terms, (to make IPLEX available ) to shut up now... . Bottom line, WHAT DO WE (PALS ) HAVE TO LOSE? --

If our (PALS & supporters) current letter writing campaign, is able to inspire, motivate, and or raise awareness to the plight of PALS. I will definitely feel like --Mission Accomplished!! Even if we are able to get the FDA to immediately change, there bogus position, I will still feel that victory is just around the corner, this fight will not be over... not by a long shot!!

If whatever we're able to accomplish with our current e-mail/ letter writing campaign helps the next generation of PALS dream bigger, work harder and once and for all, finish the work we've started, I'll always believed in my heart that we have made our mark as true champions.

But Don't Get Me Wrong, I Want to Win This Thing ASAP... If It's Wrong and or Selfish to Want to Live Longer, well then,... I'm Wrong, & Selfish -- End of Story.

Monday, February 9, 2009

Scientists Heartened at Prospect of End to Stem Cell BanBy Amanda GardnerHealthDay ReporterMONDAY, Feb. 9 (HealthDay News) -- Researchers are rejoicing over President Barack Obama's anticipated lifting of the eight-year ban on embryonic stem cell research imposed by his predecessor, President George W. Bush.The anticipation moved one step closer to reality Thursday, with media reports that Obama gave House Democrats at a closed-door Virginia retreat a "guarantee" that he would sign an executive order overturning Bush's policy."It's going to remove an embarrassment for American science," said Dr. Darwin Prockop, director of the Texas A&M Health Science Center College of Medicine Institute for Regenerative Medicine at Scott & White Hospital in Temple. "It's a statement that we're going to again believe in science."Yet those same experts are aware that the sobering state of the economy could impose its own restrictions on this type of research."This clearly is a very important part of our medical future," said Paul Sanberg, distinguished professor of neurosurgery and director of the University of South Florida Center of Excellence for Aging and Brain Repair in Tampa. "[But] to clear the path for this without giving additional money to the National Institutes of Health will be disappointing. I hope the stimulus package also includes an increase in embryonic stem cell funding."Sanberg also expressed concern that any monies redirected to stem cell research could divert funds from other critical avenues of research. "If it's a normal competitive process, it will take money away from other programs," he said.Stem cell research received a big boost in January, when the U.S. Food and Drug Administration approved the first-ever human trial using embryonic stem cells as a medical treatment.Geron Corp., a California-based biotech company, was given the OK to implant embryonic stem cells in eight to 10 paraplegic patients who can use their arms but can't walk.In 2001, then-president Bush limited federal funding for stem cell research only to human embryonic stem cell lines that already existed.The decision prompted some scientists to worry that the United States would fall behind other countries in the drive to unlock the potential of stem cell research.Embryonic stem cells are the most basic human cells, believed to be capable of growing into any type of cell in the body. Working as a sort of repair system for the body, they can theoretically divide without limit to replenish other cells. The scientific hope is that stem cells may, at some point in the future, become capable of treating a variety of diseases and conditions, such as Parkinson's disease, diabetes, heart disease and spinal cord injuries, according to the U.S. National Institutes of Health.National polls continue to find that the majority of Americans favors embryonic stem cell research, although some surveys have found that that support has declined somewhat in recent years.Many people object to the use of embryonic stem cells, contending that the research requires the destruction of potential life, because the cells must be extracted from human embryos.The stem cells being used in the recently approved Geron trial were obtained from one of the Bush administration's approved stem cell lines. And no federal funds were used in the development of this treatment.Since the restrictions on embryonic stem cell research took effect, many research institutions have redirected their focus to other types of stem cells. Prockop's institution, for instance, deals only with adult stem cells.Adult stem cells can give rise to all the specialized types of cells found in tissue from which they originated, such as skin. But, scientists don't agree on whether adult stem cells may yield cell types other than those of the tissue from which they originate, according to the National Institutes of Health.Prockop said embryonic stem cells "are mainly of interest as a research tool and a biological experimental system. Their use in patients in spite of that recent approval for Geron is really very questionable because of the potential for tumors."Still, the anticipated lessening of restrictions by the Obama administration may help funnel more private money into stem cell research, the experts said."This should give more general acceptance to stem cell research, because now, there won't be this stigma associated with it as much," Sanberg said.And, perhaps, a new federal policy would spur organizations such as the American Heart Association -- which currently does not fund research involving human embryonic stem cells or stem cells derived from fetal tissue -- to channel funds into this line of research, Sanberg added. (The heart association said it "recognizes the value of all types of stem cell research and supports federal funding of this research.")Still, Sanberg pointed out, some ethical issues surrounding stem cell research and its application will remain.For instance, he said, "There still needs to be some oversight on the uses of stem cells and cloning."More informationTo learn more about stem cells, visit the U.S. National Institutes of Health.